“Real world evidence” and “Real World Data” (RWE and RWD) have been bouncing around for several years as concepts to accelerate both the development of new drugs, and the acceptance of them by payers for coverage. However, there is considerable confusion over their status in the regulatory approval process, as well as uncertainties on their validity medically. A new Syneos Health survey, Real World Value: Advancing Payer Understanding of RWE in Rare Disease, suggests that drug sponsors need to do a better job of educating the life sciences community on RWE, especially in such instances as “single-arm” trials (which have limited trial subjects), or so-called “N-of-1” studies, where (usually) one or a few patients are getting an experimental treatment. N-of-1 results have been crucial to the development of the latest cellular and genetic therapies (CGTs).
“Our research reveals that payers are positively disposed to use RWE, but knowledge gaps around the validity and value of RWE must be closed to improve access for rare disease patients,” said Alistair Macdonald, CEO. The survey included 64 respondents, evenly divided between the US and Europe (France, Germany, Italy, Spain, Sweden and the UK). All participants were on Pharmacy and Therapeutic (P&T) committees, have experience in rare disease, are involved in decision making for this category and also formulate medical policies, says the company.
A key distinction is made in the Syneos report: Many people (whether on P&T committees or not) understand RWE to be post-approval data that verifies research claims—which is important and does influence ongoing decisionmaking—but RWE has become an important element in the data submitted by sponsors for drug approvals. This use has been developing at FDA and other regulators for a number of years, and got spurred ahead by the 21st Century Cures Act, passed in the US in 2016.
The report highlights differences between US and European approaches to RWE; generally, its understanding is broader in Europe. One startling difference: While 42% of European respondents consider sponsor dossiers “always meaningful,” only 16% of US respondents do. The sponsor dossier is the materials a drug company submits to P&T committees for formulary decisions; the skepticism on the US side speaks to the lack of trust in the data.
A related point is that sponsors are now allowed (by FDA rules) to provide “healthcare economic information” (HCEI) to payers as part of the dossier; however, not only are payers not aware of the shift, but many sponsors are not aware of it either. “Even among manufacturers, and especially at large multinationals with internal rare disease teams, there may be poor literacy about the changing rules of the road,” says Meg Alexander, managing director of reputation & risk management at Syneos.
Syneos offers a number of concluding recommendations, including participating in effort to standardize terminologies and lexicons around RWE and RWD; being more up-front about the structure of projects like single-arm trials; and sponsoring more patient registries, which are a way to standardize data across larger patient populations.
The Syneos report is available here.